Why is PQA focusing on rare disease?
There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments. In the United States, rare disease affects about one in 10 people. The burden and cost of treating rare disease is high for patients, our care system and society.
The economic burden of rare disease reached nearly $1 trillion in 2019, and the average drug approved under the Orphan Drug Act costs nearly $120,000 per year.
Ensuring the quality of medication use is critically important for rare disease patients, payers, clinicians, specialty pharmacies and everyone with a role in the care process. Like all patients, individuals with rare disease want medications that are accessible, safe, effective and help them achieve the best outcomes possible.
The challenges of improving medication use quality in rare disease is similar to that of conditions that affect small populations, but are not strictly defined as rare. The insights and approaches to addressing one will benefit both. Variations within type 1 diabetes, for example, often lead to small populations when consider the origins, progression and treatment of disease.
About PQA Convenes
PQA Convenes brings together national leaders in medication use quality to build consensus and develop plans of action to promote innovative and timely opportunities for improving patient care and outcomes. A gathering of diverse thought leaders and decision makers, PQA Convenes is designed to:
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Explore how medication use quality and pharmacist-provided care can improve care delivery, patient and provider experiences, and patient outcomes.
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Clarify unmet market needs, gaps in care, or interventions that can be realized through research, education, and collaboration.
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Provide a collective call to action, which can include (a) white papers or consensus statements; (b) follow-up or expanded convenings; and (c) communications and engagement strategies to build broader awareness.